Treatment of small vessel primary CNS vasculitis in children: an open-label cohort study

Refractory Primary Central Nervous System Vasculitis of Childhood: Successful Treatment with Infliximab

“…This immunosuppressive regimen led to complete neurological recovery in the majority of children 3 . There is limited knowledge of the approach to children who fail standard therapy.…”

“…We reported the efficacy and safety of a treatment protocol for SV-cPACNS 3 . This immunosuppressive regimen led to complete neurological recovery in the majority of children 3 .…”

Treatment of CNS Vasculitis in Children

“…Refractory seizure status is a well-recognized presentation of small vessel vasculitis and mandates a rapid evaluation. Blood inflammatory markers are commonly raised in children with small vessel cPACNS; the elevation may be modest though [10]. vWF antigen strongly supports a vasculitic etiology as opposed to demyelinating or other conditions; 90 % of children have abnormal CSF studies at diagnosis including increased protein level and/or leukocytosis [16].…”

“…Each subtype has a distinct treatment approach. All treatment regimens are based on observational cohorts or case reports; no clinical trials have been conducted in primary CNS vasculitis [10,11].…”

“…Angiography-negative, small vessel cPACNS is the most challenging subtype due to its clinical heterogeneity, the exponential risk of severe brain injury due to frequently disease-associated seizures, the lack of specific biomarkers, and the need for invasive brain biopsies to confirm the diagnosis [6,10]. Disease and treatment result in a very high burden for patients and families.…”